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Ivacaftor in People with CF age 2 to 5 years with a CFTR Gating Mutation (VX 770-108)

Study Type:

Interventional

Intervention Category:

CFTR Modulation

Study Sponsor:

Vertex

Study Phase:

3

Recruitment Status:

Trial Completed

Study Drug(s):

Kalydeco

Number of Participants Being Recruited:

28

Single / Multi-Center:

Multi-Center

STUDY BACKGROUND INFORMATION:

This is an open-label study designed to look at the safety, pharmacokinetics (PK), pharmacodynamics(PD), and effectiveness of ivacaftor in people with CF who are 2 through 5 years of age and have a CFTR gating mutation in at least 1 allele. Part A is designed to look at the safety and PK of multiple-dose administration of ivacaftor in children 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to look at the safety, PK, PD, and efficacy of ivacaftor in children 2 through 5 years of age.

Ivacaftor is currently approved by the US FDA and the EMA under the trade name KalydecoÂ® for the treatment of cystic fibrosis in patients 6 years of age and older who have the G551D-CFTR mutation

ELIGIBILITY

Age:

2 Years - 5 Years

FEV1:

Not Applicable

P. aeruginosa status:

Not applicable

B. cepacia status:

Not applicable

Other Primary Eligibility Requirements:

Eligible subjects must have a CFTR Gating Mutation in at least one allele

Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific
trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.

The sites listed below are currently recruiting subjects. Please note that this list generally lists either the pediatric or adult program but not both even though both programs may be recruiting subjects. Also, this list does not include sites that may be participating in the study but are not yet open for recruitment. If you are interested in this study please contact your center for more information.